Reform, Regulation, and Pharmaceuticals — The Kefauver–Harris Amendments at 50 (New Eng Jour Med)

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Reform, Regulation, and Pharmaceuticals — The Kefauver–Harris Amendments at 50

Jeremy A. Greene, M.D., Ph.D., and Scott H. Podolsky, M.D.
N Engl J Med 2012; 367:1481-1483, October 18, 2012

Fifty years ago this month, President John F. Kennedy signed into law the Kefauver–Harris Amendments to the Federal Food, Drug, and Cosmetic Act (see photoPresident John F. Kennedy Signing the 1962 Kefauver–Harris Amendments.). With the stroke of a pen, a threadbare Food and Drug Administration (FDA) was given the authority to require proof of efficacy (rather than just safety) before approving a new drug — a move that laid the groundwork for the phased system of clinical trials that has since served as the infrastructure for the production of knowledge about therapeutics in this country. We often remember the Kefauver–Harris Amendments for the thalidomide scandal that drove their passage in 1962. But there is much we have collectively forgotten about Senator Estes Kefauver (D-TN) and his hearings on administered prices in the drug industry. Many parts of the bill left on Congress's cutting-room floor in 1962 — and left out of our memories since — have not disappeared but continue to confront those who would ensure access to innovative, safe, efficacious, and affordable therapeutics.

By the time Kefauver began his investigation into the pharmaceutical industry in the late 1950s, the escalating expense of lifesaving prescription drugs was illustrating that the free-market approach to medical innovation had costs as well as benefits. From the development of insulin in the 1920s, through the “wonder drug” revolutions of sulfa drugs, steroids, antibiotics, tranquilizers, antipsychotics, and cardiovascular drugs in the ensuing decades, the American pharmaceutical industry had come to play a dominant role in the public understanding of medical science, the economics of patient care, and the rising politics of consumerism. For Kefauver, the “captivity” of the prescription-drug consumer in the face of price gouging and dubious claims of efficacy under-scored the need for the state to ensure that innovative industries worked to the benefit of the average American.

After 17 months of hearings, in which pharmaceutical executives were openly berated for profiteering and doctors were portrayed as dupes of pharmaceutical companies' marketing departments, Kefauver presented his bill, S.1552. Perhaps its least controversial components were its calls for ensuring that the FDA review claims of efficacy before drug approval, monitor pharmaceutical advertising, and ensure that all drugs had readable generic names. More radically, Kefauver proposed completely overhauling the relationship between patents and therapeutic innovation. First, he proposed a compulsory licensing provision so that all important new drugs would generate competitive markets after 3 years. Second, and more controversial still, Kefauver wanted to eliminate “me-too drugs” and “molecular modifications” by insisting that a new drug be granted a patent only if it produced a therapeutic effect “significantly greater than that of the drug before modification.”1 Proving that a drug worked, according to Kefauver, was not enough: he wanted proof that a drug worked better than its predecessors. In contemporary terms, he wanted to know its comparative effectiveness.

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Renewed attention to comparative effectiveness research in the 21st century illustrates the consequences of sidelining Kefauver's initial demand for comparative data for evaluating the promotion of novel therapeutics. By 2000, pharmaceutical expenditures had become one of the fastest-growing parts of the budget of many U.S. states and third-party insurers. But the kind of knowledge required for entry into the U.S. drug market offers consumers and payers little information relevant to choosing between subtly different “me-too” drugs within the same therapeutic class — whose therapeutic effect may or may not be the same. Only in the past decade, through the action of the Reforming States Group, the Drug Effectiveness Review Project, and most recently funding of comparative effectiveness research through the American Recovery and Reinvestment Act, the Affordable Care Act, and now the Patient-Centered Outcomes Research Institute, have we begun to catch up on the vital project of comparing therapeutics so that American consumers and their physicians can make meaningful treatment decisions — the project that motivated Kefauver's original investigations a half century ago.

http://www.nejm.org/doi/full/10.1056/NEJMp1210007?query=health-policy-and-reform